Conjugation of a brain-penetrant peptide with neurotensin provides antinociceptive properties

J Clin Invest. 2014 Mar;124(3):1199-213. doi: 10.1172/JCI70647. Epub 2014 Feb 17.

Abstract

Neurotensin (NT) has emerged as an important modulator of nociceptive transmission and exerts its biological effects through interactions with 2 distinct GPCRs, NTS1 and NTS2. NT provides strong analgesia when administered directly into the brain; however, the blood-brain barrier (BBB) is a major obstacle for effective delivery of potential analgesics to the brain. To overcome this challenge, we synthesized chemical conjugates that are transported across the BBB via receptor-mediated transcytosis using the brain-penetrant peptide Angiopep-2 (An2), which targets LDL receptor-related protein-1 (LRP1). Using in situ brain perfusion in mice, we found that the compound ANG2002, a conjugate of An2 and NT, was transported at least 10 times more efficiently across the BBB than native NT. In vitro, ANG2002 bound NTS1 and NTS2 receptors and maintained NT-associated biological activity. In rats, i.v. ANG2002 induced a dose-dependent analgesia in the formalin model of persistent pain. At a dose of 0.05 mg/kg, ANG2002 effectively reversed pain behaviors induced by the development of neuropathic and bone cancer pain in animal models. The analgesic properties of ANG2002 demonstrated in this study suggest that this compound is effective for clinical management of persistent and chronic pain and establish the benefits of this technology for the development of neurotherapeutics.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Analgesics / chemical synthesis
  • Analgesics / pharmacokinetics
  • Analgesics / pharmacology*
  • Animals
  • Blood-Brain Barrier / metabolism
  • Bone Neoplasms / pathology
  • Capillary Permeability
  • Cell Line
  • Cell Line, Tumor
  • Chronic Pain / drug therapy
  • Drug Evaluation, Preclinical
  • Formaldehyde
  • Inhibitory Concentration 50
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Neoplasm Transplantation
  • Neuralgia / chemically induced
  • Neuralgia / drug therapy
  • Nociception / drug effects*
  • Peptides / chemical synthesis
  • Peptides / pharmacokinetics
  • Peptides / pharmacology*
  • Rats
  • Rats, Sprague-Dawley
  • Succinimides / chemical synthesis
  • Succinimides / pharmacokinetics
  • Succinimides / pharmacology*

Substances

  • ANG2002
  • Analgesics
  • Peptides
  • Succinimides
  • Formaldehyde